Speakers

Patricia Dickson, MD, Division Chief, Genetics and Genomic Medicine, Professor of Genetics, Washington University in St. Louis

More is better: enzyme replacement for lysosomal storage disorders

Jonathan Cooper, PhD, Professor of Pediatrics, Genetics and Genomic Medicine, Washington University in St. Louis

One and done? Gene therapy for lysosomal storage disorders

There are over seventy fatal inherited diseases that kill because the lysosome, the cell’s waste disposal and recycling center, stops working properly. This is because of mutations in a gene that should make a vital part of the lysosomal machinery. In theory, this should be simple to repair either by giving back what is missing (enzyme replacement therapy) or by providing a working copy of the faulty gene that encodes the enzyme (gene therapy). We shall discuss the pluses and minuses of each approach, where we are in bringing these treatments to the clinic, and how these treatments may be commercialized.

On the third Thursday of every month , ICTS Precision Medicine hosts a session featuring WashU faculty working in translational genomics/precision medicine areas. Talks are typically oriented toward biotechnology, diagnostics, informatics, start-ups and precision interventions based on medical genomics innovations.